NEOSTeM (NEOnatal STroke Mesenchymal Stem Cell) Trial: Assessing the Safety of Mesenchymal Stem Cells for Babies with Stroke

Abstract

Neonatal stroke occurs in up to 1 in 2,500 live births and can be defined as disturbances in the blood supply to the brain and includes an acute ischemic event. Up to 60% of babies diagnosed with stroke will have atypical neurological development and brain injury, including epilepsy and cerebral palsy. Current standard of care focusses on diagnosis and prompt stabilisation of symptoms such as seizures. There are no specific interventions to protect the developing brain or prevent the progression of injury. However, stem cells, particularly mesenchymal stem cells (MSCs), are a potential treatment for neonatal stroke, where early therapy may minimise lifelong impairment.

This Phase I dose escalation trial assesses the safety of early and repeated systemic (intravenous) administration of MSCs for neonatal stroke at both 2- and 5-million cells/kg body weight.